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NMDP BioTherapies, an organization offering solutions for companies developing and commercializing cell and gene therapies, and Cryoport (NASDAQ: CYRX), a leading global provider of innovative [...]

Two gene therapy products, Casgevy and Lyfgenia, have been approved by the FDA for the treatment of patients 12 years and older with sickle cell anemia. The FDA approved Casgevy to Vertex [...]

Researchers based at the University of Toronto Leslie Dan Faculty of Pharmacy have developed a novel ionizable lipid nanoparticle (LNP), called iso-A11B5C1, that can deliver mRNA specifically to [...]

Around the world, more than 2,000 gene therapies are in development, according to the American Society of Gene and Cell Therapy, which are financially attractive to drug companies. It seems the [...]

Gene therapy by using CAR-T cells, one of the most advanced methods for treating cancer, has reached the final stages of development thanks to the tireless efforts of professors at Tehran [...]

Diabetes researchers have logged another major milestone in their efforts to get people off injected insulin. The first clinical trial in humans reported the “initial success” of [...]

A minimally invasive method holds promise for the treatment of neurological disorders and injury. Korean researchers have devised a way to remotely direct stem cells to specific areas of the [...]

This guidance provides FDA’s current thinking on determining sameness of human gene therapy  products under FDA’s orphan drug regulations for the purpose of orphan-drug designation and [...]

Regenerative neuroscience aims to stimulate endogenous repair in the nervous system to replace neurons lost from degenerative diseases. Recently, we reported that overexpressing the transcription [...]

Surgeons in New York have successfully attached a kidney grown in a genetically altered pig to a human patient and found that the organ worked normally, a scientific breakthrough that one day may [...]